Souped-Up CRISPR Gene Editor Replicates and Spreads Like a Virus

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Researchers at UC Berkeley have developed a new gene editing system called NANITE that combines the precision of CRISPR-Cas9 with the replicating and disseminating capabilities of viruses. Early results show NANITE can achieve roughly three times the editing efficiency of standard CRISPR-Cas9, significantly lowering levels of disease-causing proteins in lab tests and animal models. This breakthrough addresses a key challenge in gene therapy by increasing the percentage of edited cells needed to overcome genetic disorders.

Why it matters

Traditional gene editing tools are limited by their 'one-and-done' nature, only editing the cells they directly reach. NANITE's virus-like delivery system creates a cascading effect, amplifying the editing process far beyond the initial treatment area. This could make gene editing safer and more feasible for tissues and organs that are currently difficult to target, unlocking the potential to treat a wider range of genetic diseases.

The details

The NANITE system uses virus-like proteins to encapsulate and deliver CRISPR machinery. Once inside a cell, NANITE instructs the cell to manufacture more of the CRISPR tool and package it for delivery to surrounding cells. In lab-grown cells, NANITE demonstrated roughly three times the editing efficiency of standard CRISPR-Cas9. In mice with a genetic metabolic disorder, NANITE significantly lowered levels of a harmful protein, while the original CRISPR version showed little effect at the same dosage.

• The initial tests focused on the liver, a relatively accessible organ for gene therapy.
• Researchers injected NANITE directly into the rodents' veins, a technique that shows promise in human applications.

What’s next

The team is likewise exploring converting the NANITE system to leverage mRNA delivery, which has a well-established track record thanks to its use in COVID-19 vaccines.